The OneGene Program®

The OneGene Program is a team of highly trained and dedicated people who help ensure that every family has the support they need during their treatment journey and beyond. Their work starts the moment the ZOLGENSMA® (onasemnogene abeparvovec-xioi) Prescription Form and/or Patient Consent Form is received. From that point forward, the team works closely with your family to do everything from answering questions to being a reassuring voice to speak to when things might feel overwhelming.

Contact the OneGene Program at
855-441-GENE (4363), Monday-Friday
(8 AM-8 PM ET), to learn more. Translation services available upon request.

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The OneGene Program®

The OneGene Program is a team of highly trained and dedicated people who help ensure that every family has the support they need during their treatment journey and beyond. Their work starts the moment the ZOLGENSMA® (onasemnogene abeparvovec-xioi) Prescription Form and/or Patient Consent Form is received. From that point forward, the team works closely with your family to do everything from answering questions to being a reassuring voice to speak to when things might feel overwhelming.

Contact the OneGene Program at
855-441-GENE (4363), Monday-Friday
(8 AM-8 PM ET), to learn more. Translation services available upon request.

Support available to every family includes:

A Patient Resource Manager icon

Patient Resource Manager

A Patient Resource Manager is your main point of contact on behalf of Novartis Gene Therapies, providing expert guidance and answering questions about ZOLGENSMA as your child goes through the treatment journey.

A Case Coordinator icon

Case Coordinator

A Case Coordinator acts as a go-between to connect you to resources you may need and assists doctors with insurance processes. Other support includes verification of insurance benefits and coordination of financial assistance programs for eligible patients.

Learn how the OneGene Program can help throughout the treatment journey when you watch the Starting ZOLGENSMA video.

I am passionate about helping families living with SMA because I care deeply about supporting families as they face the realities of a rare disease diagnosis. I want to ease your burden in any way I can. I am dedicated to helping you as we face your challenges together. Karen, Patient Resource Manager

It was amazing to have the OneGene Program reach out so quickly because we felt like we had someone in our corner every step of the way.

Nicole, mother of Brady

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • ZOLGENSMA can cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • ZOLGENSMA can cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.
  • Contact the patient’s doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, or if the patient misses a dose of the corticosteroid or vomits it up.

What should I watch for before and after infusion with ZOLGENSMA?

  • Viral respiratory infections before or after ZOLGENSMA infusion can lead to more serious complications. Contact the patient’s doctor immediately if you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
  • Decreased platelet counts could occur following infusion with ZOLGENSMA. Seek immediate medical attention if the patient experiences unexpected bleeding or bruising.
  • Thrombotic microangiopathy (TMA) has been reported to occur approximately one week after ZOLGENSMA infusion. Caregivers should seek immediate medical attention if the patient experiences any signs or symptoms of TMA, such as unexpected bruising or bleeding, seizures, or decreased urine output.

What do I need to know about vaccinations and ZOLGENSMA?

  • Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.
  • Protection against respiratory syncytial virus (RSV) is recommended.

Do I need to take precautions with the patient’s bodily waste?

Temporarily, small amounts of ZOLGENSMA may be found in the patient’s stool. Use good hand hygiene when coming into direct contact with bodily waste for 1 month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.

What are the possible or likely side effects of ZOLGENSMA?

The most common side effects that occurred in patients treated with ZOLGENSMA were elevated liver enzymes and vomiting.

Indication

What is ZOLGENSMA?
ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into a vein. ZOLGENSMA was not evaluated in patients with advanced SMA.

The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away.

You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or Novartis Gene Therapies, Inc. at 833-828-3947.

Please see the Full Prescribing Information.